In a significant step forward for hematological cancer treatment, Genmab has initiated a first-in-human trial of GEN3018, an investigational antibody with target and mechanism currently undisclosed. The Phase I clinical trial will focus on patients with relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS).
The open-label, multicenter study will enroll up to 78 participants across two distinct trial components. Part 1 will focus on dose escalation, systematically testing increasing doses of GEN3018 to establish safety parameters and identify optimal dosing strategies. Part 2 will refine the dosing approach based on initial safety and preliminary efficacy data. Primary endpoints include evaluating dose-limiting toxicities and overall adverse events, with secondary objectives spanning pharmacokinetic analysis, immunogenicity assessment, and preliminary anti-tumor activity measurements.
Research context
Current treatment landscapes for relapsed/refractory AML and HR-MDS remain challenging. Standard therapeutic approaches, including intensive chemotherapy and hypomethylating agents, often yield limited long-term outcomes. Recent innovations like venetoclax combinations and targeted molecular inhibitors have incrementally improved treatment paradigms, but significant unmet medical needs persist. Recent innovative approvals include:
- Venetoclax combination therapies
- FLT3 inhibitors (midostaurin, gilteritinib)
- IDH inhibitors (ivosidenib, enasidenib)